Ivosidenib for IDH1-Mutant Intrahepatic Cholangiocarcinoma: Insights From a Multicenter Real-World Study.

doi:10.1111/liv.70295

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Article

Ivosidenib for IDH1-Mutant Intrahepatic Cholangiocarcinoma: Insights From a Multicenter Real-World Study.

Liver international : official journal of the International Association for the Study of the Liver - 1 Sept 2025

Niger Monica, Rimini Margherita, Castet Florian, Melzer Anna, Rizzato Mario Domenico, Pressiani Tiziana, Lavacchi Daniele, Aprile Giuseppe, Angela Torsello, Saladino Tiziana, Silvia Noventa, Cito Pasqua, Pastorino Alessandro, Terán-Brage Eduardo, Sciortino Carolina, Camera Silvia, Pircher Chiara, Persano Mara, Mazzaferro Vincenzo, Foti Silvia, Vega Kreina Sharela, Ettrich Thomas J, Antonuzzo Lorenzo, Rimassa Lorenza, Lonardi Sara, Perkhofer Lukas, Macarulla Teresa, Pietrantonio Filippo, Casadei-Gardini Andrea

Abstract excerpt

BACKGROUND & AIMS: Cholangiocarcinoma (CCA) is a rare cancer with limited therapeutic options and a poor prognosis. While first-line combination therapies have improved outcomes, second-line treatment remains challenging. Ivosidenib, an IDH1 inhibitor, has shown promise in treating IDH1 mutant CCA, but real-world data is limited. This study aims to evaluate ivosidenib's efficacy and safety in a large cohort of...

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Identifiers

PMID: 40856270
DOI: 10.1111/liv.70295
PMCID: PMC12379572

Genes

IDH1

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